EA201991309A1 - Способы модуляции сплайсинга рнк - Google Patents

Способы модуляции сплайсинга рнк

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Publication number
EA201991309A1
EA201991309A1 EA201991309A EA201991309A EA201991309A1 EA 201991309 A1 EA201991309 A1 EA 201991309A1 EA 201991309 A EA201991309 A EA 201991309A EA 201991309 A EA201991309 A EA 201991309A EA 201991309 A1 EA201991309 A1 EA 201991309A1
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Eurasian Patent Office
Prior art keywords
methods
rems
another aspect
gene
intronic
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EA201991309A
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English (en)
Russian (ru)
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EA201991309A 2016-11-28 2017-11-27 Способы модуляции сплайсинга рнк EA201991309A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662426619P 2016-11-28 2016-11-28
PCT/US2017/063323 WO2018098446A1 (en) 2016-11-28 2017-11-27 Methods for modulating rna splicing

Publications (1)

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EA201991309A1 true EA201991309A1 (ru) 2019-11-29

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EA201991309A EA201991309A1 (ru) 2016-11-28 2017-11-27 Способы модуляции сплайсинга рнк

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US (2) US11702646B2 (cg-RX-API-DMAC7.html)
EP (1) EP3544435A4 (cg-RX-API-DMAC7.html)
JP (2) JP2019535789A (cg-RX-API-DMAC7.html)
CN (1) CN110352007A (cg-RX-API-DMAC7.html)
AU (1) AU2017363369A1 (cg-RX-API-DMAC7.html)
CA (1) CA3043755A1 (cg-RX-API-DMAC7.html)
EA (1) EA201991309A1 (cg-RX-API-DMAC7.html)
MX (1) MX2019005588A (cg-RX-API-DMAC7.html)
WO (1) WO2018098446A1 (cg-RX-API-DMAC7.html)

Families Citing this family (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4249472A3 (en) 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Methods for modulating rna splicing
CN110352007A (zh) 2016-11-28 2019-10-18 Ptc医疗公司 用于调节rna剪接的方法
CN111372611A (zh) 2017-06-14 2020-07-03 Ptc医疗公司 修饰rna剪接的方法
IL272413B (en) 2017-08-04 2022-09-01 Skyhawk Therapeutics Inc Methods and compositions for modulating splicing
EP3833357A4 (en) * 2018-08-07 2022-06-08 The Children's Hospital of Philadelphia REGULATION OF GENE EXPRESSION BY ALTERNATIVE SPLICING, AND THERAPEUTIC METHODS
WO2020076892A1 (en) * 2018-10-09 2020-04-16 The University Of North Carolina At Chapel Hill Regulated gene editing system
CN114007613A (zh) 2019-02-05 2022-02-01 斯基霍克疗法公司 用于调节剪接的方法和组合物
EP3920928A4 (en) 2019-02-06 2022-09-28 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATION OF SPLICING
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
US20220307053A1 (en) 2019-07-25 2022-09-29 Novartis Ag Regulatable expression systems
WO2021034985A1 (en) * 2019-08-19 2021-02-25 Stoke Therapeutics, Inc. Compositions and methods for modulating splicing and protein expression
IL293340A (en) 2019-12-02 2022-07-01 Storm Therapeutics Ltd Polyheterocyclic compounds as mettl3 inhibitors
AU2021218807A1 (en) * 2020-02-12 2022-09-15 The Children's Hospital Of Philadelphia Compositions and methods for inducible alternative splicing regulation of gene expression
IL295955A (en) 2020-02-28 2022-10-01 Remix Therapeutics Inc Compounds and methods for modulating splicing
EP4110775A1 (en) 2020-02-28 2023-01-04 Remix Therapeutics Inc. Thiophenyl derivatives useful for modulating nucleic acid splicing
KR20220157407A (ko) 2020-02-28 2022-11-29 레믹스 테라퓨틱스 인크. 핵산 스플라이싱을 조절하기 위한 피리다진 유도체
WO2021174164A1 (en) 2020-02-28 2021-09-02 Remix Therapeutics Inc. Compounds and methods for modulating splicing
CN116134036A (zh) 2020-04-08 2023-05-16 雷密克斯医疗公司 用于调节剪接的化合物和方法
JP2023520916A (ja) 2020-04-08 2023-05-22 リミックス セラピューティクス インコーポレイテッド スプライシングを調整するための化合物及び方法
AU2021272972A1 (en) 2020-05-13 2022-12-08 Chdi Foundation, Inc. HTT modulators for treating Huntington's disease
KR20230051168A (ko) 2020-07-02 2023-04-17 레믹스 테라퓨틱스 인크. 2-(인다졸-5-일)-6-(피페리딘-4-일)-1,7-나프티리딘 유도체, 및 핵산 스플라이싱의 조절제로서의 및 증식성 질환의 치료를 위한 관련 화합물
KR20230118067A (ko) 2020-07-02 2023-08-10 레믹스 테라퓨틱스 인크. 5-[5-(피페리딘-4-일)티에노[3,2-c]피라졸-2-일]인다졸 유도체, 및 핵산 스플라이싱의 조절제로서의 및 증식성 질환의 치료를 위한 관련 화합물
WO2022214520A1 (en) * 2021-04-07 2022-10-13 Janssen Sciences Ireland Unlimited Company Antibacterial compounds
CN113267626B (zh) * 2021-05-17 2023-01-13 郑州大学第一附属医院 一种用于高危人群贲门腺癌早期筛查的试纸条
CN113604566B (zh) * 2021-07-13 2023-05-16 中山大学孙逸仙纪念医院 lncRNA BCYRN1在膀胱癌预后、治疗中的应用
US20240409536A1 (en) 2021-08-30 2024-12-12 Remix Therapeutics Inc. Compounds and methods for modulating splicing
EP4395892A1 (en) 2021-08-30 2024-07-10 Remix Therapeutics Inc. Compounds and methods for modulating splicing
US20240400584A1 (en) 2021-08-30 2024-12-05 Remix Therapeutics Inc. Compounds and methods for modulating splicing
US20240368163A1 (en) 2021-08-30 2024-11-07 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023034836A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023064879A1 (en) 2021-10-13 2023-04-20 Remix Therapeutics Inc. Compounds and methods for modulating nucleic acid splicing
US20250332173A1 (en) 2021-10-13 2025-10-30 Remix Therapeutics Inc. Compounds and methods for modulating nucleic acid splicing
JP2024540477A (ja) 2021-11-17 2024-10-31 シーエイチディーアイ ファウンデーション，インコーポレーテッド ハンチントン病を治療するためのｈｔｔモジュレータとしてのｎ－（２ｈ－インダゾール－５－イル）ピラジン－２－カルボキサミド誘導体及び類似化合物
WO2023133229A2 (en) 2022-01-05 2023-07-13 Remix Therapeutics Inc. Compounds and methods for modulating splicing
TW202346305A (zh) 2022-01-05 2023-12-01 美商雷密克斯醫療公司 用於調節剪切之化合物及方法
WO2023133217A1 (en) 2022-01-05 2023-07-13 Remix Therapeutics Inc. 2-(indazol-5-yl)-6-(piperidin-4-yl)-1,7-naphthyridine derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases
CN118647405A (zh) * 2022-01-25 2024-09-13 上海魁特迪生物科技有限公司 改善认知障碍的方法
EP4568962A2 (en) * 2022-08-09 2025-06-18 PTC Therapeutics, Inc. Methods for modulating rna splicing

Family Cites Families (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3558618A (en) 1968-04-01 1971-01-26 Dow Chemical Co Novel 4h-pyrazino(1,2-a)pyrimidine-4-ones
US4122274A (en) 1977-05-25 1978-10-24 Bristol-Myers Company 3-Tetrazolo-5,6,7,8-substituted-pyrido[1,2-a]pyrimidin-4-ones
JPS56150091A (en) 1980-03-28 1981-11-20 Janssen Pharmaceutica Nv 3-(1-piperidinylalkyl)-4h-pyrido(1,2-a)pyrimidine- 4-one derivative and its manufacture
US4342870A (en) 1980-03-28 1982-08-03 Janssen Pharmaceutica N.V. Novel 3-(1-piperidinylalkyl)-4H-pyrido[1,2-a]pyrimidin-4-one derivatives
US5089633A (en) 1987-04-28 1992-02-18 Georgia Tech Research Corporation Substituted isocoumarins
US5599816A (en) 1990-05-02 1997-02-04 Abbott Laboratories Quinolizinone type compounds
AU4231293A (en) 1992-05-13 1993-12-13 E.I. Du Pont De Nemours And Company Substituted pyrido(1,2-A)pyrimidinone derivatives as fungicides
JPH11510478A (ja) 1995-06-06 1999-09-14 アボツト・ラボラトリーズ キノリジノン型化合物
US5869500A (en) 1996-12-13 1999-02-09 Hoffmann-La Roche Inc. Pyridone compounds useful in treating Alzheimer's disease
EP1115724A1 (en) 1998-09-21 2001-07-18 Shire Biochem Inc. Quinolizinones as integrin inhibitors
DE60024861T2 (de) 1999-10-28 2006-07-06 Trine Pharmaceuticals, Inc., Waltham Pumpeninhibitoren zur freisetzung von medikamenten
CN1891699A (zh) 2000-01-24 2007-01-10 基纳西亚股份有限公司 用于治疗的吗啉代基取代的化合物
WO2002053576A1 (en) 2001-01-05 2002-07-11 The General Hospital Corporation Viral delivery system for infectious transfer of large genomic dna inserts
US20050009843A1 (en) 2001-04-26 2005-01-13 Kiyoshi Nakayama Medicine for inhibiting drug elimination pump
JP2005516005A (ja) 2001-12-07 2005-06-02 バーテクス ファーマスーティカルズ インコーポレイテッド Ｇｓｋ−３阻害剤として有用なピリミジンベースの化合物
GB0205281D0 (en) 2002-03-06 2002-04-17 Novartis Ag Organic compounds
EP1542667B1 (en) 2002-07-24 2012-03-07 PTC Therapeutics, Inc. Ureido substituted benzoic acid compounds and their use for nonsense suppression and the treatment of disease
US9068234B2 (en) 2003-01-21 2015-06-30 Ptc Therapeutics, Inc. Methods and agents for screening for compounds capable of modulating gene expression
ES2339862T3 (es) 2003-06-20 2010-05-26 Novartis Vaccines And Diagnostics, Inc. Compuestos de piridino 1,2-a-pirimidin-4-ona como agentes anticancerosos.
US20080255162A1 (en) 2004-05-04 2008-10-16 Warner-Lambert Company Llc Pyrrolyl Substituted Pyrido[2,3-D]Pyrimidin-7-Ones and Derivatives Thereof as Therapeutic Agents
CA2595477A1 (en) 2005-01-21 2006-07-27 Janssen Pharmaceutica N.V. Novel heterocyclic benzo[c]chromene derivatives useful as modulators of the estrogen receptors
EP1874791A4 (en) * 2005-04-29 2009-01-07 Univ North Carolina METHODS AND COMPOSITIONS FOR REGULATED EXPRESSION OF NUCLEIC ACID AT POST-TRANSCRIPTIONAL LEVEL
US7563601B1 (en) 2005-06-01 2009-07-21 City Of Hope Artificial riboswitch for controlling pre-mRNA splicing
CA2612389A1 (en) * 2005-06-17 2006-12-28 Genizon Biosciences, Inc. Genemap of the human genes associated with longevity
EP1948803A4 (en) 2005-10-13 2009-11-25 Bc Cancer Agency MODULAR GENOME FOR SYNTHETIC BIOLOGY AND METABOLISM MANIPULATION
AR059339A1 (es) 2006-02-09 2008-03-26 Chugai Pharmaceutical Co Ltd Derivados de la cumarina para trastornos proliferativos de celulas, composicion farmaceutica y agente terapeutico que los contiene
US8110681B2 (en) 2006-03-17 2012-02-07 The United States Of America As Represented By The Secretary, Department Of Health And Human Services Compounds for the treatment of spinal muscular atrophy and other uses
WO2008013997A2 (en) 2006-07-27 2008-01-31 The Trustees Of Columbia University In The City Of New York Fluorescent substrates for monoamine transporters as optical false neurotransmitters
EP2114903B1 (en) 2006-12-22 2011-01-26 Avexa Limited Bicyclic pyrimidinones and uses thereof
US8633019B2 (en) 2008-05-27 2014-01-21 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
EP2138493A1 (en) 2008-06-26 2009-12-30 Sanofi-Aventis Substituted pyrimidone derivatives
PL2212324T3 (pl) 2008-07-02 2014-11-28 Avexa Ltd Związki posiadające właściwości przeciwwirusowe
WO2010019236A1 (en) 2008-08-13 2010-02-18 Ptc Therapeutics, Inc Methods for treating spinal muscular atrophy
WO2010083338A2 (en) * 2009-01-14 2010-07-22 Philadelphia Health And Education Corporation Modulation of pre-mrna using splice modulating oligonucleotides as therapeutic agents in the treatment of disease
US20100303776A1 (en) 2009-04-16 2010-12-02 The University Of North Carolina At Chapel Hill Methods and compositions for regulated expression of multiple nucleic acids
WO2011050245A1 (en) 2009-10-23 2011-04-28 Yangbo Feng Bicyclic heteroaryls as kinase inhibitors
WO2011062853A1 (en) 2009-11-20 2011-05-26 Merck Sharp & Dohme Corp. Quinolizidinone carboxamide m1 receptor positive allosteric modulators
BR112012017473A2 (pt) 2010-01-13 2019-09-24 Institut National De La Sante Et De La Rech Medicale Inserm Epst pirido(1,2-a) pirimidinas, seu uso, bem como composição farmacêutica compreendendo tais compostos
WO2012047487A1 (en) 2010-09-27 2012-04-12 Evolva Sa 2-pyridone antimicrobial compositions
WO2013059606A1 (en) 2011-10-21 2013-04-25 Tufts Medical Center, Inc. Compounds and methods for the treatment of muscular disease, and related screening methods
WO2013101974A1 (en) 2011-12-30 2013-07-04 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
CN104203239B (zh) 2012-01-26 2017-09-08 Ptc医疗公司 用于治疗脊髓性肌萎缩的化合物
ES2697174T3 (es) * 2012-02-10 2019-01-22 Ptc Therapeutics Inc Compuestos para tratar atrofia muscular espinal
US9371336B2 (en) 2012-03-01 2016-06-21 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
MX358514B (es) 2012-03-23 2018-08-24 Ptc Therapeutics Inc Compuestos para tratar la atrofia muscular espinal.
US9212209B2 (en) 2012-07-13 2015-12-15 Indiana University Research And Technology Corporation Screening methods for spinal muscular atrophy
US9040712B2 (en) 2013-01-23 2015-05-26 Novartis Ag Thiadiazole analogs thereof and methods for treating SMN-deficiency-related-conditions
CA2915764A1 (en) 2013-08-19 2015-02-26 F. Hoffmann-La Roche Ag Screening method
WO2015095446A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount of rna transcripts
US10195202B2 (en) 2013-12-19 2019-02-05 Ptc Therapeutics, Inc. Methods for modulating the amount of RNA transcripts
WO2015095449A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount rna transcripts
RS59718B1 (sr) * 2014-05-15 2020-01-31 Hoffmann La Roche Jedinjenja za lečenje spinalne mišićne atrofije
WO2016042015A1 (en) * 2014-09-16 2016-03-24 Centre National De La Recherche Scientifique (Cnrs) Method for evaluating developmental competence of an oocyte
EP3256126B1 (en) * 2015-02-09 2024-03-27 F. Hoffmann-La Roche AG Compounds for the treatment of cancer
CN107635999B (zh) 2015-05-20 2020-09-25 豪夫迈·罗氏有限公司 用于治疗脊髓性肌萎缩的化合物
EP4249472A3 (en) 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Methods for modulating rna splicing
CN110352007A (zh) 2016-11-28 2019-10-18 Ptc医疗公司 用于调节rna剪接的方法
CN111372611A (zh) 2017-06-14 2020-07-03 Ptc医疗公司 修饰rna剪接的方法
IL272413B (en) 2017-08-04 2022-09-01 Skyhawk Therapeutics Inc Methods and compositions for modulating splicing
WO2019060917A2 (en) 2017-09-25 2019-03-28 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR SCREENING AND IDENTIFYING SPREADING MODULATORS

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